Treatment of Severe Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation
Status:
Completed
Trial end date:
2007-10-01
Target enrollment:
Participant gender:
Summary
This protocol was a prospective, Phase I study of allogeneic bone marrow transplantation
(BMT) as the primary therapy for Osteogenesis Imperfecta Types II and III. Compatible sibling
donors and unrelated donors were stratified and analyzed according to the type of donor. All
patients with a sibling donor will received a chemotherapy conditioning regimen; a non-T cell
depleted allogeneic marrow, and GVHD prophylaxis. All patients with an unrelated donor will
receive a chemoradiotherapy conditioning regimen, a T-cell depleted allogeneic marrow, and
GVHD prophylaxis. The primary objective of this study was to investigate the safety and
toxicity of these BMT procedures in this particular population.